Last updated: September 15, 2026
The FDA approved Scholar Rock’s Isembyld (apitegromab) for spinal muscular atrophy 19 days ahead of its September 30 action date, while detailed DESTINY-Lung04 data at WCLC showed Enhertu stretching first line PFS to 14.3 months in HER2 mutant lung cancer alongside an immature overall survival question.
This page covers the Isembyld approval and the SAPPHIRE data behind it, the full DESTINY-Lung04 and TAISHAN-302 results from WCLC 2026 in Seoul, Definium’s third pivotal win, Corbus’s CB1 obesity data, the Novo rebrand, and Monday’s market closes.
Why did the FDA approve Scholar Rock’s Isembyld early?
The FDA approved Isembyld (apitegromab-mstn) on Friday evening, September 11, for spinal muscular atrophy in adults and children two years and older who are on SMN2 targeted treatment. The action date was September 30, making the approval 19 days early by our arithmetic.
Scholar Rock announced the approval in a press release issued Friday at 6:25 pm Eastern, and the news surfaced broadly in trade coverage on Monday. Isembyld is a fully human IgG4 monoclonal antibody that inhibits myostatin, and it is the first muscle targeted treatment approved in SMA. It is dosed at 10 mg/kg by intravenous infusion once every four weeks, layered on top of either nusinersen or risdiplam. The label covers 5q SMA patients already receiving SMN2 targeted therapy, which makes it an add on franchise rather than a replacement one.
The early action is notable given the file’s history. In August, Scholar Rock removed Catalent’s Indiana site from the application after an inspection issue and substituted an alternate fill finish facility, a change that looked like a delay risk at the time and that forced withdrawal of the European application. Instead, the American review finished ahead of schedule. The approval also earned Scholar Rock a rare pediatric disease priority review voucher. The company says product ships within days. No list price was disclosed in the announcement, and Scholar Rock shares fell 6.4% on Monday, the first session after the Friday evening release. Market data, no cause assigned.
What did the SAPPHIRE trial show for apitegromab in SMA?
SAPPHIRE randomized 188 patients aged 2 to 21 across nine countries and met its primary endpoint: a 2.2 point improvement on the Hammersmith Functional Motor Scale Expanded versus SMN2 therapy alone at about one year, with p=0.0121.
| SAPPHIRE (Phase 3, double blind, placebo controlled) | Isembyld + SMN2 therapy | Placebo + SMN2 therapy |
|---|---|---|
| Primary: HFMSE change vs control at ~1 year | +2.2 points, p=0.0121 | |
| Patients with ≥3 point HFMSE gain | 34.2% | 13.5% (odds ratio 3.8, p=0.0125) |
| Fracture rate | 9% | 2% |
| Continued into ONYX open label extension | 98% | |
The safety database spans more than 500 individuals across the apitegromab program, with some patients treated for over seven years. The most common adverse reactions were upper respiratory infections, vomiting, cough, viral infections, headache, gastroenteritis, pharyngitis, and hypersensitivity reactions. The fracture imbalance is the one number worth watching in the postmarketing period for a drug whose entire purpose is preserving motor function.
What does the early approval mean for the FDA’s decision queue?
Scholar Rock was one of four companies we have tracked whose reviews sat in what we called the queue: files moving through an FDA working under new permanent leadership, with several action dates extended by major amendments. Isembyld is the first of them to resolve, and it resolved early.
| Sponsor / program | Action date | Status |
|---|---|---|
| Scholar Rock, apitegromab (SMA) | September 30, 2026 | Approved September 11, 19 days early |
| Ultragenyx, UX111 (Sanfilippo syndrome type A) | September 19, 2026 | Pending, decision due Friday |
| Summit, ivonescimab (NSCLC) | November 14, 2026 | Pending, extension risk flagged |
| Capricor, deramiocel (DMD cardiomyopathy) | November 22, 2026 | Pending, after major amendment |
| Exelixis, zanzalintinib (mCRC) | March 3, 2027 per multiple trade outlets; no company release yet | Extended from December 3 by a major amendment, per trade reports |
One early approval does not make a pattern, but it is the first hard evidence in months that the queue can move faster than its stated dates, not just slower. The next test is Friday.
What did Enhertu’s DESTINY-Lung04 trial show in first line HER2 mutant lung cancer?
Enhertu delivered a median progression free survival of 14.3 months versus 8.3 months for pembrolizumab plus chemotherapy in first line HER2 mutant advanced non small cell lung cancer, a hazard ratio of 0.63, in detailed data presented at the WCLC 2026 Presidential Symposium in Seoul.
AstraZeneca and Daiichi Sankyo released the full numbers on September 13. DESTINY-Lung04 enrolled 454 patients with unresectable, locally advanced or metastatic nonsquamous NSCLC carrying HER2 exon 19 or exon 20 mutations, randomized one to one against platinum and pemetrexed plus pembrolizumab. It is the first Phase 3 trial to beat the global standard of care on progression free survival in this setting.
| DESTINY-Lung04 (n=454) | Enhertu 5.4 mg/kg | Chemo + pembrolizumab |
|---|---|---|
| Median PFS (primary) | 14.3 months | 8.3 months (HR 0.63; 95% CI 0.50 to 0.79; p<0.0001) |
| Objective response rate | 70.0% | 44.5% |
| Median duration of response | 13.4 months | 9.7 months |
| Interim overall survival (46.9% mature) | 29.3 months | 33.1 months (HR 1.15; 95% CI 0.88 to 1.52) |
| ILD / pneumonitis, all grades | 20.8% (grade 5: 1.8%) | Not comparable, see release |
Why is the overall survival number in DESTINY-Lung04 getting attention?
Because at the interim analysis, with survival data 46.9% mature, median overall survival numerically favored the control arm, 33.1 months versus 29.3, with a hazard ratio of 1.15 whose confidence interval spans 0.88 to 1.52. The trial continues to a final overall survival analysis.
An immature interim with a wide interval is not evidence of harm, and crossover and post progression therapy will complicate any final read. But the companies printed the number anyway, alongside an interstitial lung disease rate of 20.8% that includes 1.8% fatal events. That combination, a six month PFS gain sitting next to an overall survival curve that currently leans the wrong way and a real pneumonitis toll, is exactly the surrogate versus survival tension this market has been repricing all month. Readers of our September 8 and 9 coverage will recognize the mechanism. What is different here is the disclosure posture: everything is on the table, uncomfortable numbers included.
How do the two B7-H3 ADCs compare in relapsed small cell lung cancer?
Hansoh’s risvutatug rezetecan and MediLink’s tambotatug pelitecan posted an identical hazard ratio for death, 0.46, in separate Chinese Phase 3 trials against topotecan, but with different medians: 18.5 versus 10.3 months for the Hansoh drug, 13.3 versus 9.4 for MediLink’s.
Roche, which holds worldwide rights to tambotatug pelitecan (Tam-Peli, YL201) outside mainland China, Hong Kong, and Macau, published the TAISHAN-302 detail on September 13 with a simultaneous New England Journal of Medicine publication. GSK holds ex Greater China rights to Hansoh’s risvutatug rezetecan (HS-20093), whose ARTEMIS-008 data led the same congress’s Presidential Symposium.
| Relapsed SCLC vs topotecan | ARTEMIS-008 (risvutatug rezetecan, GSK ex China) | TAISHAN-302 (tambotatug pelitecan, Roche ex China) |
|---|---|---|
| Patients (China only) | 461 | 451 |
| Median OS | 18.5 vs 10.3 months | 13.3 vs 9.4 months |
| OS hazard ratio | 0.46 (0.35 to 0.62) | 0.46 (0.35 to 0.62) |
| Median PFS | 7.2 vs 3.0 months | 7.4 vs 2.8 months (HR 0.29) |
| Response rate | 58.3% vs 12.6% | 59.1% vs 9.7% |
| Grade ≥3 TRAEs | 60.9% vs 78.2% | 46.4% vs 74.7% |
| Global Phase 3 | Reads out 2027 | Roche plans rapid initiation |
The identical hazard ratios and near identical confidence intervals across two independent trials are about as clean a class validation as oncology produces. The median gap comes partly from different censoring and follow up, and the comparator arms behaved differently, 10.3 versus 9.4 months on the same control drug. Both readouts are China only populations, so the working assumption we published for GSK’s global program, a hazard ratio somewhere between the Chinese result and a still approvable 0.65, now applies to Roche’s as well. TAISHAN-302 adds one differentiated card: intracranial progression free survival of 6.1 versus 4.2 months and an intracranial response rate of 32.4% versus 2.9% in brain metastasis patients, plus a lower grade 3 toxicity burden than either the control arm or the rival ADC.
Did Amgen present DeLLphi-305 data at WCLC 2026?
No. As of Monday night US time, with the congress in its final day in Seoul, no efficacy figures from DeLLphi-305 had been presented or published. Amgen’s September 8 release remains the only disclosure: endpoints met, no numbers.
The scope of that statement: our checks Monday night covered Amgen’s newsroom, the WCLC program listing, where the DeLLphi-305 entry is a trial in progress poster, and congress coverage from multiple trade outlets. Amgen has said detailed data will come at an upcoming congress. The company presents at a Morgan Stanley conference Tuesday at 11:30 am Eastern, an appearance we have flagged since Friday as carrying two disclosure tests, one on the olpasiran cardiovascular outcomes timeline and one, as of this week, on tarlatamab language. A relapsed setting competitor printed an 8 month median survival gain at this congress and a first line maintenance winner printed nothing.
What did Definium’s Panorama study show in generalized anxiety disorder?
DT120 ODT 100 µg beat placebo by 5.1 points on the Hamilton Anxiety Rating Scale at week 12, p<0.0001, effect size 0.64, replicating the 5.4 point result from the Voyage study. It is the third pivotal win for the LSD derived program.
Panorama randomized 245 adults across 32 centers. The separation appeared at week one, 5.3 points, and on day two on clinician global severity. Response rate was 32% versus 14%, remission 15% versus 4%. Safety again showed no suicidality signal; the most common adverse event was transient illusion on dosing day at 68%, with discontinuation rates similar across arms. Definium plans a pre NDA meeting in the fourth quarter and an NDA filing in the first half of 2027. Shares rose 3.5% Monday. For the Voyage background, see our August analysis of the Voyage data.
What did Corbus report for CRB-913 in obesity?
CRB-913, an oral peripherally restricted CB1 inverse agonist, produced 5.0% placebo adjusted weight loss at the top dose over 12 weeks in the Phase 1b CANYON-1 study, with no serious or severe psychiatric adverse events among 188 dosed participants.
| CANYON-1, week 12 (n=254) | Placebo | 20 mg | 40 mg | 60 mg |
|---|---|---|---|---|
| Mean weight loss | 0.0% | 2.8% | 3.3% | 5.0% (all p<0.0001) |
| Patients losing ≥5% | 44.4% |
The number that matters is not the weight loss, which is modest against incretins, but the neuropsychiatric line: one transient moderate depressive event across all treated participants, in the mechanism class that produced rimonabant’s withdrawal and that Novo shelved monlunabant over. Irritability at the top dose was 9.7%, diarrhea ran 21.5% to 26.2%, and discontinuations for adverse events ranged up to 13.1%. Corbus positions the drug for combination with incretins and takes the data to ObesityWeek in November, with Phase 2 in the first half of 2027. Shares traded nearly four times Friday’s volume and closed down 0.5%. The same day, Eli Lilly discontinued an early stage obesity candidate that Fierce Biotech reported failed to clear the company’s internal bar, a reminder of how high that bar now sits.
What else happened in biotech and pharma on September 14, 2026?
Novo Nordisk rebranded its day to day operating name to Novo, retiring “Nordisk” from everyday use while keeping the legal entity Novo Nordisk A/S, the Apis bull logo, and introducing a culture program it calls The Novo Way. GSK said it will wind down its 115 year old Dresden, Germany flu vaccine plant by 2027, with Reuters reporting 641 jobs at risk, citing falling flu vaccine demand. Electra Therapeutics set terms for a Nasdaq IPO that Reuters reports targets a $977.6 million valuation; trade outlets differ on the exact raise, so we are not printing a total. Cellectis discontinued its allogeneic CAR T programs to refocus on in vivo cell therapy, per Fierce Biotech, the second such pivot this month after TScan’s. The FDA is examining how it sets criteria and endpoints for rare disease trials, per Endpoints News. Boston Scientific discontinued its Imager II angiographic catheters after manufacturing issues, per MedTech Dive. And BioNTech detailed a survival benefit for its anti CTLA-4 candidate gotistobart ahead of a pivotal readout, per Endpoints News.
How did biotech and pharma stocks close on September 14, 2026?
Exchange closes, market data, no cause assigned unless a disclosure is noted. AstraZeneca’s move followed both the Saturday SERENA-4 disclosure and the Sunday DESTINY-Lung04 presentation; Friday’s close predated both.
| Ticker | Sept 14 close | Move | Context |
|---|---|---|---|
| AZN | $163.78 | +2.3% | First session after the SERENA-4 miss printed; the miss did not price |
| SRRK | $51.85 | −6.4% | First session after Friday evening approval; list price undisclosed |
| AMGN | $381.50 | +1.1% | Four session slide ends the day before its Morgan Stanley appearance |
| AMLX | $33.64 | +6.4% | Eleventh consecutive close below the $35.50 offer; close still unannounced |
| RARE | $13.61 | −4.8% | UX111 decision due Friday |
| DFTX | $40.25 | +3.5% | Panorama win |
| CRBP | $8.08 | −0.5% | Heavy volume on CANYON-1 data |
| CGEM | $20.40 | −0.9% | Second session after REZILIENT3 |
| BSX | $45.05 | +4.8% | No cause assigned; October 28 guidance watch stands |
| GILD | $146.41 | +1.9% | |
| NVS | $138.99 | +1.3% | |
| NVO | $43.45 | +0.9% | Rebrand day |
| BMY | $64.10 | +0.7% | |
| MRK | $144.85 | +0.6% | |
| EXEL | $56.21 | +0.2% | Still no company release on the March 3 date |
| BHVN | $12.64 | −0.6% |
Frequently asked questions
What is Isembyld and who is it approved for?
Isembyld (apitegromab-mstn) is a myostatin inhibiting monoclonal antibody from Scholar Rock, approved for adults and children two years and older with 5q spinal muscular atrophy who are currently receiving SMN2 targeted treatment such as nusinersen or risdiplam.
When was Isembyld approved?
The FDA approved it on September 11, 2026, announced in a company release at 6:25 pm Eastern that Friday evening, 19 days ahead of the September 30 action date. The 19 day figure is our arithmetic from the two disclosed dates.
How is Isembyld given?
By intravenous infusion at 10 mg/kg once every four weeks, on top of ongoing SMN2 targeted therapy. It does not replace nusinersen or risdiplam.
What is Isembyld’s list price?
Scholar Rock did not disclose a price in its approval announcement, and no price had been published as of Monday night, September 14. The company says product ships within days.
Why did Scholar Rock stock fall after the approval?
Shares fell 6.4% on Monday, the first session after the Friday evening announcement. We do not assign causes to market moves without a disclosure; the undisclosed launch price and a run up ahead of approval are both context, not confirmed causes.
What is the difference between Enhertu’s PFS and OS results in DESTINY-Lung04?
Progression free survival strongly favored Enhertu, 14.3 versus 8.3 months, hazard ratio 0.63. Interim overall survival, only 46.9% mature, numerically favored the control arm at 33.1 versus 29.3 months, hazard ratio 1.15 with a confidence interval of 0.88 to 1.52. The survival analysis continues.
Is Enhertu approved in HER2 mutant lung cancer already?
Enhertu has held US accelerated approval in previously treated HER2 mutant NSCLC since 2022. DESTINY-Lung04 is the trial designed to move it into first line use against the pembrolizumab based standard.
What is a B7-H3 ADC?
An antibody drug conjugate that targets B7-H3, a surface protein overexpressed in small cell lung cancer and other tumors, and delivers a cytotoxic payload directly to those cells. Two of them, Hansoh’s risvutatug rezetecan and MediLink’s tambotatug pelitecan, have now each cut the risk of death 54% versus topotecan in Chinese Phase 3 trials in relapsed small cell lung cancer.
Who has rights to the two B7-H3 ADCs outside China?
GSK licensed risvutatug rezetecan (HS-20093) from Hansoh with rights outside Greater China, with a global Phase 3 reading out in 2027. Roche holds worldwide rights to tambotatug pelitecan outside mainland China, Hong Kong, and Macau, and says it plans to rapidly start global Phase 3 trials.
Has Amgen released DeLLphi-305 numbers for tarlatamab?
No. As of Monday night, September 14, Amgen had disclosed only that the trial met overall survival, progression free survival, and response endpoints at an interim analysis. No medians, hazard ratios, or response rates have been published, and the data were not presented at WCLC 2026.
What did Definium’s Panorama trial show?
DT120 ODT 100 µg reduced the Hamilton Anxiety Rating Scale score by 9.8 points versus 4.7 for placebo at week 12, a 5.1 point difference, p<0.0001, with benefit visible at week one and no suicidality signal. It replicates the Voyage study’s 5.4 point result.
When could Definium file DT120 with the FDA?
The company plans a pre NDA meeting in the fourth quarter of 2026 and an NDA submission in the first half of 2027, backed by what it describes as four complementary positive studies.
Why does Corbus’s 5% weight loss matter if GLP-1 drugs do better?
Because it came from a peripherally restricted CB1 inverse agonist with essentially clean neuropsychiatric safety, one transient depressive event among 188 dosed, in a mechanism class abandoned twice over psychiatric risk. The commercial thesis is oral combination therapy and maintenance, not monotherapy competition with incretins.
What changed in Novo’s name?
The company now operates day to day as Novo, with a new tagline and culture program. The legal entity remains Novo Nordisk A/S, and the Apis bull logo stays.
What are the next catalysts to watch this week?
Amgen and Amylyx both present at Morgan Stanley on Tuesday, September 15. Ultragenyx’s UX111 decision is due Friday, September 19. Grail’s advisory committee briefing documents are expected around September 21 ahead of the September 23 panel.
Sources
Primary sources (September 11 to 14, 2026): Scholar Rock press release, September 11 (FDA approval of Isembyld, SAPPHIRE data); AstraZeneca and Daiichi Sankyo press release, September 13 (DESTINY-Lung04 detailed results); Roche press release, September 13 (TAISHAN-302 detailed results, NEJM publication); Definium Therapeutics press release, September 14 (Panorama topline); Corbus Pharmaceuticals press release, September 14 (CANYON-1 topline); Novo Nordisk announcement, September 14 (rebrand); Amgen press release, September 8 (DeLLphi-305 topline, no figures); Hansoh Pharma press release, September 13 (ARTEMIS-008).
Trade coverage (September 14, 2026): Fierce Pharma (Scholar Rock approval coverage; GSK Dresden closure; Novo rebrand); Fierce Biotech (Lilly obesity discontinuation; Cellectis pivot; Electra IPO); Endpoints News (FDA rare disease criteria review; gotistobart); Reuters (GSK Dresden job figure; Electra valuation); MedTech Dive (Boston Scientific Imager II discontinuation); STAT (Novo rebrand). Market data are exchange closes.
Related coverage
- AstraZeneca’s Etcamah missed its first line trial as ARTEMIS-008 cut death risk 54% (September 14)
- Why the FDA stalled Scholar Rock’s SMA filing in August 2026
- Definium’s Voyage data and what they mean for GAD treatment
- The week the surrogate discount began: pelacarsen and the Lp(a) selloff (September 8)
- BMS met its pivotal myeloma trial with the numbers withheld (September 10)
